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Journal Articles

RNA interference therapy in acute hepatic porphyrias

Makiko Yasuda, Siobán Keel, Manisha Balwani

Blood (2023) 142 (19): 1589–1599.

https://doi.org/10.1182/blood.2022018662

Published: 2023

Includes: Supplemental data

Journal Articles

Erythropoietic Protoporphyria: Phase 2 Clinical Trial Results Evaluating the Safety and Effectiveness of Dersimelagon (MT-7117), an Oral MC1R Agonist

Manisha Balwani, Herbert L. Bonkovsky, MD, Kirstine J Belongie, PhD, Karl E Anderson, MD, Fumihiro Takahashi, PhD, Antonio Irizarry, MBA, Mark Amster, MD, D. Montgomery Bissell, MD, Bruce Wang, MD, Lydie Hazan, MD, Charles J. Parker, MD, Edward Cordasco, MD, Cynthia Levy, MD, Robert J. Desnick, MD PhD

Blood (2020) 136 (Supplement 1): 51.

https://doi.org/10.1182/blood-2020-142467

Published: 2020

Journal Articles

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

David J. Kuter, MD, Sioban Keel, MD, Charles J. Parker, MD, David C. Rees, MD, Ulrich Stölzel, MD, Paolo Ventura, MD, Manisha Balwani, Laurent Gouya, MD, PhD, Amy Simon, MD, Shangbin Liu, PhD, John J Ko, PharmD,MSc, Sean Rhyee, MD, M.P.H, Samuel M. Silver, MD PhD

Blood (2020) 136 (Supplement 1): 13.

https://doi.org/10.1182/blood-2020-134564

Published: 2020

Journal Articles

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Blood (2017) 130 (Supplement 1): 2211.

https://doi.org/10.1182/blood.V130.Suppl_1.2211.2211

Published: 2017

Journal Articles

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Timothy M. Cox, Guillermo Drelichman, Renata Cravo, Manisha Balwani, Thomas Andrew Burrow, Ana Maria Martins, Elena Lukina, Barry Rosenbloom, Ozlem Goker-Alpan, Nora Watman, Amal El-Beshlawy, Priya S. Kishnani, Maria Lucia Pedroso, Sebastiaan J. M. Gaemers, Regina Tayag, M. Judith Peterschmitt

Blood (2017) 129 (17): 2375–2383.

https://doi.org/10.1182/blood-2016-12-758409

Published: 2017

Journal Articles

Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry

Pramod Mistry, MD PhD FRCP, Neal J. Weinreb, MD FACP, Julie Batista, ScM, ScD, Hans C. Andersson, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD, Joel Charrow, MD, Paige Kaplan, MB, BCh, Aneal Khan, MD MSc, FCCMG, FRCPC, Priya S. Kishnani, MD MB BS, Edwin H. Kolodny, MD, Barry E. Rosenbloom, MD, C. Ronald Scott, MD

Blood (2016) 128 (22): 4877.

https://doi.org/10.1182/blood.V128.22.4877.4877

Published: 2016

Journal Articles

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Eliane Sardh, MD PhD, Pauline Harper, MD PhD, Nabil Al-Tawil, MD, Manisha Balwani, MD MS, Karl Anderson, MD, Joseph Bloomer, MD, D. Montgomery Bissel, MD, Robert J. Desnick, MD, Charles J. Parker, MD, John D. Phillips, PhD, Herbert L. Bonkovsky, MD, Craig Penz, MA, Amy Chan, PhD, Chang-Heok Soh, PhD, William Querbes, PhD, Amy Simon, MD, Penelope Stein, MD PhD, David C. Rees, FRCP, FRCPath, MA, MBBS

Blood (2016) 128 (22): 2318.

https://doi.org/10.1182/blood.V128.22.2318.2318

Published: 2016

Journal Articles

Engage - a Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Eliglustat in Adults with Gaucher Disease Type 1: Results after 18 Months

Dominick J Amato, MD, Majed Dasouki, MD, Seymour Packman, MD PhD, Gregory M. Pastores, MD, Sarit Assouline, MD MSc, Manisha Balwani, MD MS, Pramod Mistry, MD PhD FRCP, Andres Ortega, MD, Suma Shankar, MD PhD, Maria Helena Solano, MD, Leorah H. Ross, MD PhD, Jennifer Angell, ScM, Judith Peterschmitt, MD

Blood (2014) 124 (21): 2732.

https://doi.org/10.1182/blood.V124.21.2732.2732

Published: 2014

Journal Articles

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

Barry E. Rosenbloom, MD, Timothy M. Cox, MD, Guillermo I. Drelichman, MD, Renata Cravo, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD FAAP, FACMG, Ana Maria Martins, MD PhD, Elena Lukina, MD PhD, Leorah H. Ross, MD PhD, Jennifer Angell, ScM, Judith Peterschmitt, MD

Blood (2014) 124 (21): 1406.

https://doi.org/10.1182/blood.V124.21.1406.1406

Published: 2014

Journal Articles

Encore: A Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat To Imiglucerase In Gaucher Disease Type 1 Patients On Enzyme Replacement Therapy Who Have Reached Therapeutic Goals

Thomas Andrew Burrow, MD, FAAP, FACMG, Manisha Balwani, MD, MS, Timothy M. Cox, MD, Guillermo Drelichman, MD, Renata Cravo, MD, Ana Maria Martins, MD, PhD, Elena Lukina, MD, PhD, Barry E. Rosenbloom, MD, Leorah H. Ross, MD, PhD, Jennifer Angell, ScM, Ana Cristina Puga, MD, PhD

Blood (2013) 122 (21): 3468.

https://doi.org/10.1182/blood.V122.21.3468.3468

Published: 2013

Journal Articles

Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results

Suma Shankar, MD, PhD, Elena Lukina, MD, PhD, Dominick J Amato, MD, Majed Dasouki, MD, Seymour Packman, MD, PhD, Gregory M Pastores, MD, Sarit Assouline, MD, Manisha Balwani, MD, MS, Pramod K Mistry, MD, PhD, FRCP, Leorah H Ross, MD, PhD, Sachin Marulkar, MBBS, MPH, M. Judith Peterschmitt, MD

Blood (2013) 122 (21): 2275.

https://doi.org/10.1182/blood.V122.21.2275.2275

Published: 2013

Journal Articles

The porphyrias: advances in diagnosis and treatment

Manisha Balwani, Robert J. Desnick

Blood (2012) 120 (23): 4496–4504.

https://doi.org/10.1182/blood-2012-05-423186

Published: 2012

NARROW

RNA interference therapy in acute hepatic porphyrias

Erythropoietic Protoporphyria: Phase 2 Clinical Trial Results Evaluating the Safety and Effectiveness of Dersimelagon (MT-7117), an Oral MC1R Agonist

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Engage - a Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Eliglustat in Adults with Gaucher Disease Type 1: Results after 18 Months

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

Encore: A Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat To Imiglucerase In Gaucher Disease Type 1 Patients On Enzyme Replacement Therapy Who Have Reached Therapeutic Goals

Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results

The porphyrias: advances in diagnosis and treatment

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American Society of Hematology

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